Study of Safety And Efficacy Of ReFacto AF In Previously Untreated Hemophilia A Patients In The Usual Care Setting
10 maja 2019 zaktualizowane przez: Pfizer
AN OPEN-LABEL STUDY OF THE SAFETY AND EFFICACY OF REFACTO AF IN PREVIOUSLY UNTREATED PATIENTS IN USUAL CARE SETTINGS
Study to evaluate the safety and effectiveness of ReFacto AF for the treatment of severe hemophilia A in patients who have not yet received treatment for their hemophilia.
Study subjects will be males less than 6 years old who have not taken any clotting factor or other blood products before the study.
The safety and effectiveness of ReFacto AF will be determined in this study by tests and procedures done at the doctor's office.
Przegląd badań
Szczegółowy opis
Regulatory Commitment
Typ studiów
Interwencyjne
Zapisy (Rzeczywisty)
23
Faza
- Faza 4
Kontakty i lokalizacje
Ta sekcja zawiera dane kontaktowe osób prowadzących badanie oraz informacje o tym, gdzie badanie jest przeprowadzane.
Lokalizacje studiów
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Nantes, Francja, 44093
- CHU de Nantes
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Madrid, Hiszpania, 28046
- Hospital Universitario La Paz
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Valencia, Hiszpania, 46009
- Hospital Universitario La Fe
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Groningen, Holandia, 9713 GZ
- University Medical Center Groningen
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Adana, Indyk, 01330
- Cukurova Universitesi Tip Fakultesi
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Istanbul, Indyk, 34098
- Istanbul Universitesi Cerrahpasa Tip Fakultesi
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Izmir
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Bornova, Izmir, Indyk, 35100
- Ege Universitesi Tip Fakultesi
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Bremen, Niemcy, 28177
- Klinikum Bremen-Mitte gGmbH, Professor Hess Kinderklinik
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Niedersachsen
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Hannover, Niedersachsen, Niemcy, 30625
- Medizinische Hochschule, Hannover
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Lviv, Ukraina, 79044
- Derzhavna ustanova "Instytut patolohii krovi ta transfuziinoi medytsyny Natsionalnoi akademii
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Firenze, Włochy, 50134
- Agenzia per L'emofilia e Centro Regionale Riferimento Coagulopatie Congenite
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Kryteria uczestnictwa
Badacze szukają osób, które pasują do określonego opisu, zwanego kryteriami kwalifikacyjnymi. Niektóre przykłady tych kryteriów to ogólny stan zdrowia danej osoby lub wcześniejsze leczenie.
Kryteria kwalifikacji
Wiek uprawniający do nauki
Nie starszy niż 6 lat (Dziecko)
Akceptuje zdrowych ochotników
Nie
Płeć kwalifikująca się do nauki
Męski
Opis
Inclusion Criteria:
- Male subjects <6 years of age with severe hemophilia A (FVIII:C <1%) based on clinical records, including newborns.
- No prior exposure to factor products or any blood products.
Exclusion Criteria:
- Presence of any bleeding disorder in addition to hemophilia A.
- Treatment with any investigational agent or device within the past 30 days.
- Any condition(s) that compromises the ability to collect study-related observations, or that poses a contraindication to study participation (these conditions include, but are not limited to, inadequate medical history to assure study eligibility; and expectation of poor adherence to study requirements).
Plan studiów
Ta sekcja zawiera szczegółowe informacje na temat planu badania, w tym sposób zaprojektowania badania i jego pomiary.
Jak projektuje się badanie?
Szczegóły projektu
- Główny cel: Inny
- Przydział: Nie dotyczy
- Model interwencyjny: Zadanie dla jednej grupy
- Maskowanie: Brak (otwarta etykieta)
Broń i interwencje
Grupa uczestników / Arm |
Interwencja / Leczenie |
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Eksperymentalny: 1
The investigator treats subjects with ReFacto AF in the usual care setting.
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Blood draws to determine the level of factor VIII activity before and after dosing at appropriate visits, and blood draws to determine levels of factor VIII inhibitor (antibody to factor VIII).
Inne nazwy:
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Co mierzy badanie?
Podstawowe miary wyniku
Miara wyniku |
Opis środka |
Ramy czasowe |
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Percentage of Participants Who Developed Clinically Significant Factor VIII (FVIII) Inhibitors During the Course of the Study
Ramy czasowe: 2 years
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Percentage of participants who developed clinically significant FVIII inhibitors: those persistent over a defined period with clinically impactful effects like breakthrough bleed, low recovery, etc., during the course of the study.
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2 years
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Miary wyników drugorzędnych
Miara wyniku |
Opis środka |
Ramy czasowe |
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Annualized Bleeding Rate (ABR)
Ramy czasowe: 2 years
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Annualized bleeding rate was calculated as the number of bleeds divided by the treatment interval duration (enrollment visit to final visit) and then multiplied by 365.25.
If there was more than 1 bleed location (like ankle and joint) with identical bleed start date and time, it was treated as 1 bleed occurrence.
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2 years
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Total Number of Infusions to Treat a New Bleed Classified on Basis of Response to First On-Demand Treatment With Refacto AF
Ramy czasowe: 2 years
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Number of infusions of Refacto AF required to treat a new bleed were classified on basis of the response to at 4-point response scale of assessment (excellent, good, moderate and no response).
Assessment was completed each time a participant experienced a new bleed requiring an 'on-demand' IV infusion.
Excellent: definite pain relief and/or improvement in bleeding signs within 8 hours (hr) after infusion, no additional infusion administered; Good: definite pain relief and/or improvement in bleeding signs within 8 hr after infusion, at least 1 additional infusion administered for complete resolution or with no additional infusion administered; Moderate: probable or slight improvement starting after 8 hr following infusion, at least 1 additional infusion administered for complete resolution; No Response: no improvement at all between infusions or during 24 hr interval following infusion or condition worsen.
Bleeds for which response not recorded, reported as: Data Not Recorded.
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2 years
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Total Number of Infusions Needed for Resolution of Bleeding Episodes Classified on Basis of Response to Study Drug Infusion
Ramy czasowe: Within 48 hours after infusion, up to 2 years treatment duration
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Number of infusions of Refacto AF required for resolution of a bleeding episodes were classified on basis of the response at 4-point response scale of assessment (excellent, good, moderate and no response).
Excellent: definite pain relief and/or improvement in bleeding signs within 8 hours (hr) after infusion, no additional infusion administered; Good: definite pain relief and/or improvement in bleeding signs within 8 hr after infusion, at least 1 additional infusion administered for complete resolution or with no additional infusion administered; Moderate: probable or slight improvement starting after 8 hr following infusion, at least 1 additional infusion administered for complete resolution; No Response: no improvement at all between infusions or during 24 hr interval following infusion or condition worsen.
Bleeds for which response not recorded, reported as: Data Not Recorded.
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Within 48 hours after infusion, up to 2 years treatment duration
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Total Number of Breakthrough Bleeding Episodes Occurring Within 48 Hours After a Prophylaxis Infusion of ReFacto AF
Ramy czasowe: 2 years
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The number of breakthrough bleeds (spontaneous or traumatic) within 48 hours following a prophylaxis dose of ReFacto AF are summarized.
If there was more than 1 bleed location (like ankle and joint) with identical bleed start date and time, it was treated as 1 bleed occurrence.
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2 years
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Consumption of Total International Units of Factor VIII
Ramy czasowe: 2 years
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2 years
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Consumption of Total International Units of Factor VIII Per Year
Ramy czasowe: 2 years
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Consumption of total international units of Factor VIII per year was calculated for a participant: dividing the total consumption of factor VIII by participant's treatment interval duration (in days), then multiplying by 365.25.
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2 years
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Mean Dose (IU) of Study Drug Consumed Per Infusion
Ramy czasowe: 2 years
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Mean dose for each participant was calculated as participant's total factor VIII consumption (in IU) divided by the number of infusions administered.
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2 years
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Consumption of Total International Units of Factor VIII by Weight
Ramy czasowe: 2 years
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Consumption of total international units of Factor VIII by weight was calculated for a participant: dividing the total consumption of factor VIII by participant's weight (the most recently recorded).
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2 years
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Consumption of Total International Units of Factor VIII Per Year by Weight
Ramy czasowe: 2 years
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Consumption of total international units of Factor VIII per year by weight was calculated for a participant: the total consumption of factor VIII divided by participant's treatment interval duration (in days), then multiplying by 365.25 and then dividing by participant's weight (the most recently recorded).
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2 years
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Mean Dose (IU) of Study Drug Consumed Per Infusion by Weight
Ramy czasowe: 2 years
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Mean dose for each participant was calculated as participant's total factor consumption (in IU) divided by the number of infusions administered and then dividing by participant's weight (the most recently recorded).
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2 years
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Mean of Total Number of Infusions of Study Drug Received
Ramy czasowe: 2 years
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2 years
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Mean of Total Number of Days Participants Exposed to Study Drug
Ramy czasowe: 2 years
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2 years
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Number of Participants Who Required Dose Escalation of Their Prescribed Prophylaxis Regimen During Their Participation in This Study
Ramy czasowe: 2 years
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The number of participants who met the dose escalation criteria were prescribed a higher dose and/or were prescribed more frequent doses.
When dose escalation was required, the specific dose and dosing schedule was at the investigator's discretion.
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2 years
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Percentage of Bleeding Episodes With Less-Than-Expected Therapeutic Effect (LETE) in On-Demand (OD) Setting
Ramy czasowe: 2 years
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LETE occurs in OD setting if participant recorded 2 successive "No Response" (no improvement at all between infusions, or condition worsens) ratings after 2 successive infusions of study drug.
Infusions must have been given within 24 hours (hr) of each other for treatment of same bleeding event in absence of confounding factors (known presence or subsequent identification of a FVIII inhibitor, known inadequate dose for type and/or severity of bleed in opinion of investigator, delay of greater than (>) 4 hr between onset of bleed to infusion, delay of >24 hr before administration of a follow-up infusion, known compromised study drug, faulty administration of study drug, participant had an underlying, predisposing condition responsible for bleed in opinion of investigator, ongoing trauma responsible for continued bleeding.
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2 years
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Percentage of Bleeding Episodes With Less-Than-Expected Therapeutic Effect (LETE) in the Prophylaxis Setting
Ramy czasowe: 2 years
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LETE in prophylaxis setting if there was a spontaneous bleed within 48 hours after a regularly scheduled prophylactic dose of study drug (which was not used to treat a bleed) in the absence of confounding factors (known presence or subsequent identification of a FVIII inhibitor, known inadequate prophylactic dose [a dose less than that prescribed in participant's regimen], known lack of adherence to the prescribed prophylaxis regimen, known compromised study drug, faulty administration of study drug, participant had an underlying, predisposing condition responsible for the bleed in the opinion of the investigator, traumatic injury responsible for bleeding.
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2 years
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Total Number of Events of Potential Less-Than-Expected Therapeutic Effect (LETE) in the Low Recovery Setting
Ramy czasowe: 2 years
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LETE was lower than expected recovery of FVIII in the opinion of the investigator following infusion of study drug in the absence of confounding factors (known presence or subsequent identification of a FVIII inhibitor, known compromised study drug, faulty administration of study drug including inadequate dosing).
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2 years
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Współpracownicy i badacze
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Publikacje i pomocne linki
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Daty zapisu na studia
Daty te śledzą postęp w przesyłaniu rekordów badań i podsumowań wyników do ClinicalTrials.gov. Zapisy badań i zgłoszone wyniki są przeglądane przez National Library of Medicine (NLM), aby upewnić się, że spełniają określone standardy kontroli jakości, zanim zostaną opublikowane na publicznej stronie internetowej.
Główne daty studiów
Rozpoczęcie studiów (Rzeczywisty)
10 lutego 2010
Zakończenie podstawowe (Rzeczywisty)
24 listopada 2016
Ukończenie studiów (Rzeczywisty)
24 listopada 2016
Daty rejestracji na studia
Pierwszy przesłany
29 lipca 2009
Pierwszy przesłany, który spełnia kryteria kontroli jakości
30 lipca 2009
Pierwszy wysłany (Oszacować)
31 lipca 2009
Aktualizacje rekordów badań
Ostatnia wysłana aktualizacja (Rzeczywisty)
19 lipca 2019
Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości
10 maja 2019
Ostatnia weryfikacja
1 maja 2019
Więcej informacji
Terminy związane z tym badaniem
Słowa kluczowe
Dodatkowe istotne warunki MeSH
Inne numery identyfikacyjne badania
- 3082B2-4434
- B1831006 (Inny identyfikator: Alias Study Number)
- 2008-008436-93 (Numer EudraCT)
Plan dla danych uczestnika indywidualnego (IPD)
Planujesz udostępniać dane poszczególnych uczestników (IPD)?
TAK
Opis planu IPD
Pfizer will provide access to individual de-identified participant data and related study documents (e.g.
protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions.
Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
Te informacje zostały pobrane bezpośrednio ze strony internetowej clinicaltrials.gov bez żadnych zmian. Jeśli chcesz zmienić, usunąć lub zaktualizować dane swojego badania, skontaktuj się z register@clinicaltrials.gov. Gdy tylko zmiana zostanie wprowadzona na stronie clinicaltrials.gov, zostanie ona automatycznie zaktualizowana również na naszej stronie internetowej .